Zafgen’s Prader-Willi syndrome therapy receives orphan drug designation in Europe

13 July 2014 (Last Updated July 13th, 2014 18:30)

The European Commission (EC) has granted orphan drug designation to US-based Zafgen for its beloranib for treating Prader-Willi syndrome.

The European Commission (EC) has granted orphan drug designation to US-based Zafgen for its beloranib for treating Prader-Willi syndrome.

Beloranib is a potent inhibitor of Methionine aminopeptidase-2 that reduces hunger while stimulating the use of stored fat as an energy source (MetAP2). MetAP2 is an enzyme that modulates the activity of key cellular processes that control metabolism.

Zafgen CEO Thomas Hughes said: "On the basis of our Phase II results reported earlier this year, we believe beloranib represents a promising new approach for the treatment of PWS, with the potential to meaningfully improve the lives of patients with this severe and life-threatening condition.

"We believe beloranib represents a promising new approach for the treatment of PWS."

"We are dedicated to the advancement of beloranib for the treatment of PWS and other severe forms of obesity and we look forward to initiating our Phase III clinical programme in PWS later this year."

Zafgen received orphan designation from the US Food and Drug Administration for beloranib in January 2013 for treatment of Prader-Willi syndrome.

Initial data from the Phase II study of beloranib in Prader-Willi syndrome patients showed improvements in hunger-related behaviours and body composition, including reductions in body fat content and preserved lean body mass.

Zafgen is exclusively licensed beloranib from South Korea-based Chong Kun Dang Pharmaceutical. The company holds exclusive worldwide rights (exclusive of South Korea) to develop and commercialise beloranib.